Mesoblast Limited · ASX:MSB
Mesoblast — Bell Potter 2018 Emerging Leaders Conference
33 slides from Mesoblast's conference presentation, Nov 7, 2018
Slide 1: Cellular Medicines for Intractable Serious and Life-Threatening Diseases
Nasdaq: MESO ASX: MSB Cellular Medicines for Intractable Serious and Life-Threatening Diseases Bell Potter 2018 Emerging Leaders Conference Sydney Australia, October 2018 Exhibit 99.4
Slide 2: Cautionary Note Regarding Forward-Looking Statements
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, perf
Slide 3: Our Mission
Our Mission Mesoblast is committed to bring to market disruptive cellular medicines to treat serious and life-threatening illnesses |
Slide 4: Premier Global Cellular Medicines Company
Premier Global Cellular Medicines Company Multiple Revenue Generating Products & Phase 3 Assets Industrial Scale Manufacturing Disruptive Technology Platform1 Immuno-selected, culture expanded cellular medicines Well characterized mechanisms of action targeting multiple pathways Extensive, robust IP
Slide 5: Commercial Translation Capabilities
Commercial Translation Capabilities Technology positioned for scalable, industrialized manufacturing Immune privileged nature of mesenchymal lineage cells enables allogeneic “off the shelf” product candidates Culture expansion scalable to produce anticipated commercial quantities Management know-how
Slide 6: Global IP Estate Provides Substantial Competitive Advantage
~800 Patents and patent applications (69 Patent families) across all major jurisdictions Covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells Enables licensing to third parties for different indications, when in alignment with our corporate strategy,
Slide 7: Commercial Products and Clinical Pipeline
IN DEVELOPMENT PLATFORM PRODUCT THERAPEUTIC AREA APPROVAL COMMERCIAL RIGHTS MSC (Bone Marrow) TEMCELL® HS Inj1 Acute GVHD Japan MSC (Adipose) Alofisel2 Perianal Fistula Global MARKETED Commercial Products and Clinical Pipeline Using Mesoblast’s Intellectual Property and Technology Platform Mesoblast
Slide 8: Diverse Pipeline of Cellular Medicines
Diverse Pipeline of Cellular Medicines
Slide 9: MSC-100-IV (remestemcel-L) for Steroid-Refractory Acute GVHD
MSC-100-IV (remestemcel-L) for Steroid-Refractory Acute Graft vs Host Disease |
Slide 10: Remestemcel-L: Market Opportunity for Acute GVHD
Minimal Treatment Options Market Opportunity Burden of Illness aGVHD is a life-threatening complication that occurs in ~50% of patients receiving allogeneic bone marrow transplants (BMT)1 Steroid-refractory aGVHD is associated with mortality rates as high as 95%1 and significant extended hospital st
Slide 11: Remestemcel-L: Product Development Strategy
Remestemcel-L: Product Development Strategy | Target pediatric patients with SR-aGVHD first Extensive safety and efficacy data generated and published in children with SR-aGVHD1,2 High economic burden in treatment of children with SR-aGVHD Fast-track designation provides pathway for priority review
Slide 12: Remestemcel-L: Phase 3 Trial Operational Update
Remestemcel-L: Phase 3 Trial Operational Update | Phase 3 study evaluated remestemcel-L in 55 children to improve overall response rate and survival 89% of children had grade C/D disease, the most severe form and historically associated with up to 95% mortality Study successfully met the primary end
Slide 13: GVHD Pathway to Market
GVHD Pathway to Market Regulatory Preparations for Biologics License Application (BLA) filing underway Pre-BLA meeting targeted Q4 CY2018 Fast Track designation allows eligibility for priority review and rolling BLA review process Commercial Parallel track commercial planning for pricing, reimbursem
Slide 14: MPC-150-IM for Chronic Heart Failure
MPC-150-IM for Chronic Heart Failure |
Slide 15: Common Treatment Pathway in Progressive Heart Failure
Source: Simon-Kucher & Partners 2017. Primary research 2017; Payers n=35, KOLs n=15, Cath lab managers n=4. Corlanor® (ivabradine) approved by FDA (April 2015). ENTRESTO® (sacubitril/valsartan) approved by FDA (July 2015). GlobalData-PharmaPoint Heart Failure (2016); McMurray et al., 2012;Yancy et a
Slide 16: Heart Failure Market Opportunity
Market Opportunity Burden of Illness/Limited Options Unmet Need ~ 8 million patients with chronic heart failure by 2030 in USA alone1 17-45% globally die within 1 year of hospital admission1 Majority of advanced heart failure patients die within 5 years1 Despite recent advances in newly approved dru
Slide 17: MPC-150-IM: Phase 3 Program in Advanced Heart Failure
MPC-150-IM: Phase 3 Program in Patients with Advanced Heart Failure 85% of patients enrolled in events-driven USA Phase 3 trial, targeting approx. 600 patients Pre-specified interim futility analysis of the efficacy endpoint in the first 270 patients was successfully achieved in April 2017 In April
Slide 18: MPC-150-IM: End-Stage Heart Failure Market Opportunity
Market Opportunity Burden of Illness/Limited Options Unmet Need/ Orphan Indication MPC-150-IM: End-Stage Heart Failure Market Opportunity ~50k new patients/year in USA alone have end-stage heart failure1 1-year mortality can exceed 50% in these patients2 Only ~2K - 3K heart transplants are performed
Slide 19: The 21st Century Cures Act and RMAT Designation
The 21st Century Cures Act (Cures Act) Legislation for An Expedited Approval Path for Cellular Medicines Designated as Regenerative Medicine Advanced Therapies (RMAT) Cellular medicines may be designated as regenerative advanced therapies, if they are intended to treat, modify, reverse, or cure a se
Slide 20: INTERMACS Adverse Event Rates in LVAD Patients
*Interagency Registry for Mechanically Assisted Circulation (INTERMACS): Events per 100 Patient-Months in the First 12 Months Post-Implant, based on 7,286 patients with CF-LVADs between 2012-2014. INTERMACS* Adverse Event Rates in LVAD Patients: the most common cause of non-surgical hospitalization
Slide 21: Proposed Mechanism of Action for MPC-150-IM
Proposed mechanism of action for MPC-150-IM: reduce GI bleeding through both reduction in inflammation and strengthening of native heart function GI Bleeding Is Due To Right Ventricular Dysfunction And Engorgement of Abnormal GI Vessels GI Vascular Abnormalities (Dysplasia and Leakiness) Are Due To
Slide 22: Pilot Trial of Low-Dose MPCs in LVAD Patients
30-patient 2:1 randomized, controlled trial of 25M MPCs vs saline injection into native heart at time of LVAD MPC group had prolonged median time to first hospitalization (91 days) vs control group (51 days) 50% of MPC vs. 20% of control patients tolerated temporary LVAD wean at 90 days At 90 days,
Slide 23: Post-Hoc Analyses: Reduction in Hospitalization due to GI Bleeding
Post-Hoc Analyses of Results from Pilot Trial of Low-Dose MPCs in LVAD Patients Show Reduction in Hospitalization due to GI Bleeding1 MPC group had significantly longer time to first hospitalization due to major GI bleeding (p<0.05, Kaplan-Meier statistics) 71% reduction in number of patients with a
Slide 24: MPC-150-IM: Phase 2b Trial of High-Dose MPCs in LVAD Patients
MPC-150-IM: Phase 2b Trial of High-Dose MPCs in End-stage Heart Failure Patients with LVADs 159-patient, double-blind, placebo-controlled 2:1 randomized Phase 2b trial, evaluating safety and efficacy of a single injection of MPC-150-IM (150 million allogeneic MPCs) into the native myocardium of LVAD
Slide 25: MPC-06-ID for Chronic Low Back Pain due to Disc Degeneration
MPC-06-ID for Chronic Low Back Pain due to Disc Degeneration |
Slide 26: MPC-06-ID: Chronic Low Back Pain Market Opportunity
Minimal Treatment Options Market Opportunity Burden of Illness Unmet Need MPC-06-ID: Chronic Low Back Pain due to Degenerative Disc Disease Back pain causes more disability than any other condition1 Inflicts substantial direct and indirect costs on the healthcare system,1 including excessive use of
Slide 27: MPC-06-ID: Phase 3 Trial in Chronic Low Back Pain
MPC-06-ID: Phase 3 Trial in Patients with Chronic Low Back Pain | Phase 3 study completed enrollment in March 2018 Over 400 patients were enrolled at 48 sites across USA and Australia Patients randomized 1:1:1 to receive saline, 6-million MPCs with hyaluronic acid and 6-million MPCs without hyaluron
Slide 28: Corporate and Financial
Corporate and Financial |
Slide 29: Strategic Partnership for Cardiology in China
Tasly Pharmaceutical Group has exclusive rights and will fund all development, manufacturing and commercialization activities in China for MPC-150-IM for the treatment or prevention of chronic heart failure and MPC-25-IC for the treatment or prevention of acute myocardial infarction Mesoblast receiv
Slide 30: Strategic Financing Transactions
Strategic Financing Transactions US$75 million non-dilutive, four-year credit facility US$35 million drawn on closing in March 2018 US$15 million may be drawn on or before Q4 CY2018, and a further US$25 million on or before Q3 CY2019, as certain milestones are met Interest 9.95% per annum with inter
Slide 31: Financial Overview: Fiscal Year Ending June 30, 2018
Financial Overview: Fiscal Year Ending June 30, 2018 Year ending (US$m) June 30, 2018 June 30, 2017 $Change % Commercialization revenue 3.6 1.4 2.2 152% Milestone revenue 13.3 0.5 12.8 NM Interest revenue 0.4 0.5 (0.1) (22%) Total revenue 17.3 2.4 14.9 NM Operating and other expenses (83.3) (92.6) 9
Slide 32: Milestones
Milestones |
Slide 33: CY 2018 Corporate Milestones
CY 2018 Corporate Milestones MSC-100-IV for Acute Graft versus Host Disease Successfully met Day 28 primary end point pediatric Phase 3 trial (Q1 CY18) Day 100 survival/safety data pediatric Phase 3 trial (Q2 CY18) Day 180 survival/safety data pediatric Phase 3 trial (Q3 CY18) FDA meetings (Q4 CY18)
Mesoblast — Bell Potter 2018 Emerging Leaders Conference on 6ix